Hypoparathyroidism, Hypoparathyroidism Postprocedural, Hypoparathyroidism Post-surgical
Conditions
Keywords
hypoparathyroidism, parathyroid hormone, PTH, serum calcium, phosphorus
Brief summary
This study is being done to see if transplanting parathyroid tissue into the forearm will help hypo-parathyroid patients achieve parathyroid hormone (PTH) levels that would help normalize their serum calcium and phosphorus levels. The parathyroid tissue used in this study will come from cadaveric tissues that were donated at the time of the death of the donor(s).
Detailed description
The company that makes the parathyroid hormone NATPARA will no longer be making the product by the end of 2024 which is one of the reasons the investigators are doing this study: to see if parathyroid transplantation is another way to treat hypoparathyroidism. This procedure is investigational and one of the first times the procedure is being done in humans. It is not approved by the FDA.
Interventions
BIOLOGICALParathyroid Allotransplant
Parathyroid Allotransplant
Sponsors
Peter Stock
National Institutes of Health (NIH)
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
Only subjects who meet all of the following criteria are eligible for enrollment. 1. Subjects age 18 or older. 2. Subjects who are able to provide written informed consent and to comply with study procedures. 3. Clinical history and laboratory data compatible with HypoPT as defined by hypocalcemia and documented PTH levels either inappropriately normal or below the normal range on two occasions greater than 2 weeks apart and 12 months after surgery, requiring treatment with activated vitamin D (≥0.5 mcg calcitrol) and oral calcium (≥800mg) daily, or currently on PTH (1-84), PTH (1-34), palopegteriparatide or other recombinant parathyroid hormone replacement injections with ongoing symptomatology due to hypocalcemia and variable degree of biochemical control. 4. No history of immunodeficiency (e.g., opportunistic infections) that could be exacerbated by immunosuppression. 5. Up to date immunizations per the University of California, San Francisco (UCSF) standard of care for organ transplantation, including influenza, pneumococcal, hepatitis B, and tetanus-diptheria
Exclusion criteria
Subjects who meet any of the following criteria are not eligible for enrollment 1. Presence of donor specific anti-HLA antibodies detected by Luminex Single Antigen/specificity bead assay including weakly reactive antibodies that would not be detected by a flow cross match 2. Intolerance to any drug that will be used as part of the IS regimen. 3. Poorly controlled diabetes with an A1C of \>8%. 4. Blood Pressure (BP): systolic blood pressure (SBP) \> 140mmHg or DBP \>90 mmHg despite treatment with antihypertensive agents. If the BP is greater than 140/90 chart review and discussion with the patient will be done to establish that BP is in good control. 5. Other
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Levels of intact PTH | From baseline to week 52 (year 1) | The primary efficacy endpoint is attainment of levels of intact PTH levels that are in the normal range and at least double the baseline intact PTH level at one year following the PTG transplant from a single donor. |
| Incidence of Serious Adverse Events | From baseline to Year 1 | The primary safety endpoints are the incidence of serious adverse events related to the transplant procedure or immunosuppression, incidence of post- transplant infections and malignancies, incidence of de novo sensitization. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Dose of oral calcium supplements and calcitrol | From baseline to week 52 (year 1) | in patients on conventional Ca and calcitriol supplementation, a 50% reduction in the pre-transplant oral dose of calcium supplements and calcitrol with maintenance of stable serum calcium in the normal; |
| Dose of subcutaneous parathyroid hormone (PTH) replacement therapy | From baseline to week 52 (year 1) | for patients on subcutaneous PTH replacement therapy, a 50% reduction in the dose of PTH replacement therapy with maintenance of serum calcium in the normal target range. |
| PTH replacement therapy | From baseline to week 52 (year 1) | Discontinuation of PTH replacement therapy while maintaining serum calcium in the normal target range while taking 1000 mg or less of elemental calcium in the form of calcium supplements. |
Countries
United States
Contacts
CONTACTCrystal Loucel, MPH, MS, RN
CONTACTRodney Rogers
PRINCIPAL_INVESTIGATORPeter Stock, MD, PhD
University of California, San Francisco
Outcome results
None listed